AT A GLANCE

Summary: The mechanisms of skeletal muscle development, growth and regeneration are described. We discuss whether these processes are dysregulated in inherited muscle diseases and identify pathways that may represent therapeutic targets.

Summary: Academia-industry partnerships are important for therapeutic development. An improved understanding of the steps required for the translation of academic discoveries will be key for future clinical success in the neuromuscular field.

Summary: Personalized medicine approaches benefit from humanized animal models. Here, we outline the usefulness, caveats and considerations for generating and using these models for pre-clinical studies of Duchenne muscular dystrophy.

Summary: This Review discusses biomarkers in blood and urine linked to myonecrosis, inflammation and oxidative stress, to enhance development of therapies for DMD, and the challenges to be overcome for clinical translation.

Editors' choice: Unlike boys with DMD, the mdx:Cmah?/? mouse shows increased weight gain and more rapid bone development; therefore, its utility for studying growth and skeletal development in DMD is limited.

Summary: The mdx mouse model of Duchenne muscular dystrophy (DMD) has in vivo abnormalities in cerebellar spike firing, which could contribute to the neurological symptoms observed in individuals with DMD.

Summary: We describe a mouse model (KI-STIM1I115F) that displays the clinical hallmarks of tubular aggregate myopathy. This model provides a new opportunity to characterize the disorder and test novel therapeutic strategies.

Summary: This study reports associations between symptom onset and the microbiome, immune system and epigenetic marks, establishing a time line that may pinpoint biomarkers of ALS for earlier diagnosis and therapeutic intervention.

Other journals from The Company of Biologists

Human geneticist and cardiologist Elizabeth McNally discusses her first experiences in science and the effort we all need to make to facilitate diversity in science, while Louis Kunkel tells us about talks about his discovery of dystrophin.

DMM authors can transfer their submission to our sister journal Biology Open with just one click. An online Open Access journal, BiO also welcomes direct submissions and focuses on the swift publication of high-quality, sound research. Recent disease modelling highlights in BiO include work by Liu et al. investigating transgenic replenishment strategies targeting myocardial and non-myocardial tissues in the zebrafish. Find out more about how to transfer.

Highlighted by the Guest editors of our special issue, Grunwald and colleagues use zebrafish to investigate interactions among ryanodine receptor isotypes in muscle development and function, while Claire Wood et al. tests the mdx:Cmah−/− mouse to investigate growth and skeletal development in Duchenne muscular dystrophy.